This Day That Year: Clementia Pharma

Shares of Clementia Pharmaceuticals Inc. (CMTA) have lost more than 38% of their value over the last 1 year while the iShares NASDAQ Biotechnology Index (ETF) has gained nearly 11% during the same period.

It was on this day last year, i.e. on August 2, 2017, that Clementia debuted on the NASDAQ Global Select Market by setting a public offering at $15.00 per share.

Montreal-based Clementia is a clinical-stage company developing treatments for rare bone disorders and other diseases.

The Company's lead product candidate is Palovarotene for the treatment of fibrodysplasia ossificans progressiva and multiple osteochondromas, two ultra-rare and disabling bone diseases.

Fibrodysplasia ossificans progressiva, also called stone man syndrome, is a rare genetic disorder wherein the body's joints, muscles and ligaments become gradually replaced with bones. As new bones keep developing with age, the patient becomes entombed in a skeleton of heterotopic bone. (Bones that do not belong to the main skeleton but that regularly develop in certain organs are called heterotrophic bones).

Being an incredibly rare disorder, Stone Man Syndrome affects 1 in 2 million people worldwide. A significant indicator of Stone Man Syndrome is the presence of malformed big toes. (Source: genetics Home reference)

In May of this year, the Company reported positive data from Part B of its ongoing phase II clinical trial evaluating Palovarotene for the treatment of patients with fibrodysplasia ossificans progressiva.

According to the trial results, treatment with Palovarotene resulted in meaningful reductions in new heterotopic ossification volume (HO, bone growth in abnormal places) as measured by both 12-week flare-up imaging and 12-month whole body CT (WBCT) scan.

A phase III trial of Palovarotene in fibrodysplasia ossificans progressiva, dubbed MOVE Trial, is underway. The MOVE Trial is designed to enroll 80 patients age 4 and older, and enrollment is expected to be completed by the end of 2018, and reach the first interim analysis in mid-2019.

Another indication for which Palovarotene is being tested is multiple osteochondromas (MO), also known as multiple hereditary exostoses.

Hereditary multiple osteochondromas is a genetic condition in which people develop multiple benign (noncancerous) bone tumors that are covered by cartilage (called osteochondromas). These tumors are not present at birth, but almost all affected people develop multiple osteochondromas by the time they are 12 years old. (Source: NIH).

A phase II study evaluating the safety and efficacy of Palovarotene for the treatment of pediatric patients with multiple osteochondromas, dubbed MO-Ped trial, was initiated as recently as April of this year.

The MO-Ped Trial is designed to enroll 240 patients ranging in age from 2 to 14 years old at approximately 29 centers in 12 countries around the world.

The 12-month interim data readout from the MO-Ped Trial is expected to be in the first half of 2020, with 24-month top-line results from the study in the first half of 2021.

Palovarotene is also under preclinical testing in dry eye disease, and may move into clinical studies this year.